Addendum
Onasemnogene abeparvovec (spinal muscular atrophy) - Addendum to Commission A21-68
Report documents
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Addendum
Onasemnogene abeparvovec (spinal muscular atrophy) - Addendum to Commission A21-68
Dossier assessment
Indication:
Patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene
Result of dossier assessment:
Unchanged after addendum:
- SMA type 1: added benefit not proven.
- SMA type 2: added benefit not proven.
- SMA type 3: added benefit not proven.
- Pre-symptomatic patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene: added benefit not proven.
Additional information
Federal Joint Committee (G-BA)
2021-11-04 A G-BA decision was published.
Project details
- Project no.
- A21-128
- Commission
- Commission awarded on 29.09.2021 by the Federal Joint Committee (GBA).
- Report type
- Addendum
- Status
- Commission completed
- Department/Division
- Drug Assessment
- Topic
- Head and nerves
- Note
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If the need for additional work on a project commissioned by the GBA arises during consultations, IQWiG submits an addendum. The subsequent decides on the extent of the added benefit concludes the early benefit assessment.
Connected Projects
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Dossier assessment
Onasemnogene abeparvovec (spinal muscular atrophy) - Benefit assessment according to §35a Social Code Book V
Status: Commission completed